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East Coast Biotech Roundup: Moderna, bluebird, Blueprint

During this early spring week, it was a great time to focus on the fledgling efforts of East Coast drug hunters. A couple small companies pulled in lucrative partnerships, and two others saw their NASDAQ dreams come true. Read on, for more of the details.

—AstraZeneca inked a notably rich deal with the small biotech startup Moderna Therapeutics of Cambridge, MA, which will receive $240 million in upfront cash, plus potential further payments that could exceed $180 million, and far more if its drug candidates hit all their development goals. Moderna makes injectable forms of RNA designed to prompt cells to manufacture therapeutic proteins that act against cancer, heart disease, and other conditions. The startup has yet to put an experimental drug into clinical trials, but London-based AstraZeneca (NYSE: AZN) ponied up millions for access to 40 of Moderna’s compounds without gaining an ownership stake in the small company.

—Cambridge, MA-based Blueprint Medicines is aiming to duplicate Novartis’ success with the cancer drug imatinib (Gleevec), and it has reassembled the scientific team behind Gleevec, a treatment for chronic myeloid leukemia: — Nick Lydon, Brian Druker, and Charles Sawyers. Blueprint is taking advantage of cheaper, faster DNA sequencing technology to mine the genome for mutations that contribute to various cancer types. It has identified small molecule drug candidates to counter hundreds of those genetic abnormalities. The small biotech startup will test its experimental compounds through new partnerships announced this week with the U.K.-based Sanger Institute and Massachusetts General Hospital.

—bluebird bio, an intentionally no-caps biotechnology startup in Cambridge, MA, announced Thursday it will collaborate with Summit, NJ-based Celgene (NASDAQ: CELG) to develop cancer treatments based on its gene therapy platform. The private startup says it has industrialized methods of genetically modifying a patient’s own immune system cells called T-cells, which are then re-infused into the patient’s blood to kill cancer cells. Celgene is making an undisclosed upfront payment to bluebird, which could also net as much as $225 million per product in option fees and milestone payments, as well as co-development rights and possible royalties. Gene therapy was already in the news Thursday as the Wall Street Journal featured a similar gene therapy effort by medical researcher Renier J. Brentjens at Memorial Sloan-Kettering in New York, where T-cells were modified to attack leukemia cells.

—Brentjens’ colleague at Memorial Sloan-Kettering, David Scheinberg, is taking another tack to find new cancer-fighting drugs by aiming at targets considered “undruggable.” He and his collaborators at Emeryville, CA-based Eureka Therapeutics published encouraging results this week on their antibody, ESK1. It was designed to target the kind of internal cancer cell protein that’s usually considered inaccessible to attack by bulky antibodies that can’t get through the cell’s outer walls. In a preclinical study, ESK1 killed leukemia cells in mouse models; Scheinberg is now planning for a first clinical trial within a year, and is in discussions with potential corporate partners.

—-As such potentially lucrative ideas erupt among New York research centers and startups, the private Partnership Fund for New York City tries to make sure they lead to growing businesses inside the metropolitan area. In a recent investment this month, the fund made a loan to 3-D printing company Shapeways for development of a Long Island City plant. The 3-D printing facility could be a resource for designers of medical devices, such as dental and orthopedic implants. The fund invests money held by the New York City Partnership Foundation, the New York City Investment Fund, and the New York Small Business Ventures Funds. But Partnership Fund CEO Maria Gotsch says suitors from other regions are always trying to lure the city’s inventors away. She jokes: It’s like venture capitalists from California are standing in front of New York’s life sciences research institutions with three things: a bag of cash, a CEO, and a moving van.

—As if that wasn’t enough action, the Boston area saw two of its young biotech companies go public. Watertown, MA-based Enanta Pharmaceuticals (NASDAQ: ENTA) and Watertown, MA-based Tetraphase Pharmaceuticals (NASDAQ: TTPH) both arrived this week on the public markets, and saw their stocks go up in early trading.

Bernadette Tansey is Xconomy's San Francisco Editor. You can reach her at btansey@xconomy.com. Follow @Tansey_Xconomy

How does Bluebird achieve their genetic modifications?
zinc fingers or Talens? Or something other than these 2 preferred methods of manipulating genes?
Recent patent approvals of SGMO IP would tend to lead me to believe that SGMO has the inside track on GM therapeutics for dozens and dozens of Rare and Orphan disease “CURES”
Any thoughts out there?